dm+d

Unassigned

New Medicines

Duchenne muscular dystrophy

Information

New molecular entity
Solid GT
Solid GT

Development and Regulatory status

None
None
Phase II Clinical Trials
Yes
Yes
Oct 20FDA lifts the clinical hold from the IGNITE DMD trial. The company had provided the FDA with updated safety and functional efficacy data for all patients dosed to date. There were no additional drug-related adverse events up to 30 months post dosing. Additionally, to mitigate the risk of SEAEs, the company amended the trial protocol to include the prophylactic use of both anti-complement inhibitor eculizumab and C1 esterase inhibitor, and increasing the prednisone dose in the first month post dosing [3].
May 20Solid Biosciences receives communication from the US FDA regarding the clinical hold on the IGNITE DMD trial. Solid had previously submitted a modified clinical protocol designed to enhance patient safety, in addition to information related to improvements to its manufacturing process. The FDA in its written communication, has retained the clinical hold and requested further data and analyses relating to the company´s improved manufacturing process [3].
Oct 18FDA grants fast track status to SGT 001 for Duchenne muscular dystrophy [3].
Oct 16Has orphan drug status in EU and US. Also rare pediatric disease designation in US [3].

Category

An adeno-associated virus (AAV) based gene therapy that delivers a synthetic dystrophin gene, called microdystrophin. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilises essential associated proteins
DMD affects about 1 in 3,500 newborn males [1].
Duchenne muscular dystrophy
Intravenous infusion

Trial or other data

Jan 21Solid Biosciences re-initiates the IGNITE DMD trial [2,3].
Nov 19Solid Biosciences suspends patient enrolment in the PI/II IGNITE DMD study as the US FDA placed the study on clinical hold. Six patients had been dosed that included three patients in the first cohort at a 5E13vg/kg dose, who continue to do well and are being followed per the study protocol, whereas three patients were subsequently dosed in the second cohort at a 2E14vg/kg dose. The first two of these patients were also doing well and were being followed per study protocol. A serious adverse event (SAE) was experienced by the third patient in the 2E14 vg/kg cohort, dosed in late October, deemed related to the study drug that was characterised by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency, was fully resolved. Cytokine or coagulopathy-related abnormalities were not observed. The patient is recovering and continues to improve [3].
Dec 17PI/II IGNITE DMD study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD) starts (NCT03368742). Patients will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. 16 children aged 4 to 17 years will be recruited in the US. Primary outcomes include safety and change from baseline in microdystrophin protein in muscle biopsies; collection of these data is due to complete Apr 22 [2].